New gene therapy fixes mistakes

Existing techniques only compensate for mutations

A new type of gene therapy allows scientists to fix DNA defects directly. That’s a potentially revolutionary improvement on present gene therapy techniques, which introduce working genes to cells — but not into the genetic library itself.

“This is a major leap of the technology,” says John Rossi, a molecular geneticist at the Beckman Research Institute of City of Hope in Duarte, Calif.

This article is available only to subscribing members. Join SSP today or Log in.